Caliway Biopharmaceuticals has announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to CBL-514, a novel small molecule drug, for the treatment of Dercum's disease. This designation provides Caliway with significant clinical development support, fee reductions, and a ten-year market exclusivity period in the EU upon approval.
CBL-514 is the first drug globally to receive both Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for Dercum's disease. Caliway anticipates that these regulatory advantages from both the EMA and FDA will accelerate the clinical development of CBL-514.
Clinical Trial Results
A Phase 2 study (CBL-0201DD, NCT05387733) of CBL-514 for Dercum's disease was completed in 2023. The study demonstrated that after CBL-514 treatment, 64.5% of painful lipomas showed a dimension reduction of more than 50%, and 38.7% of painful lipomas showed complete clearance, with pain being reduced by 4.7 points (out of 10 points).
Currently, a placebo-controlled Phase 2b study (CBL-0202DD, NCT06303570) is evaluating CBL-514's efficacy in treating Dercum's disease. Subject recruitment began in August 2024, and the study results are anticipated in Q4 2025.
About Dercum's Disease
Dercum's disease is a rare disorder characterized by the development of painful lipomas, primarily located on the trunk and extremities. The pain is chronic (greater than 3 months), symmetrical, often disabling, and resistant to traditional analgesics. The etiology of Dercum's disease remains unknown, and there is currently no approved drug or recommended standard treatment.
CBL-514: A Potential First-in-Class Therapy
Given its innovative approach to fat reduction through adipocyte apoptosis, CBL-514 is the only drug in advanced clinical stages with statistically significant efficacy results. This positions CBL-514 as a potential first-in-class therapy for Dercum's disease, offering new hope to patients suffering from this rare condition. CBL-514 is a lipolysis injection that induces adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity without systemic side effects. Preclinical studies have shown that CBL-514 upregulates apoptosis mediators caspase 3 and the Bax/Bcl-2 ratio, inducing adipocyte apoptosis in vivo and in vitro.
Caliway is investigating multiple indications for CBL-514, including non-invasive subcutaneous fat reduction, Dercum's disease, and cellulite. To date, 521 subjects have been enrolled in CBL-514 clinical studies, with 317 having received CBL-514 injection treatment. Results from eight completed clinical studies across three indications have demonstrated precise efficacy with a favorable safety profile. A global Pivotal Phase 3 study for non-surgical fat reduction is expected to begin in 2025.
